The Collaborative Improvement and Innovation Network for Children With Medical Complexity.

In response to a number of emerging issues, in 2017 the federal Maternal and Child Health Bureau funded its first collaborative quality improvement network aimed at improving the quality of life for children with medical complexity, the well-being of their families and the cost-effectiveness of their care. This paper is intended as a brief introduction to the Collaborative Improvement and Innovation Network to Advance Care for Children with Medical Complexity or CMC CoIIN project. In it, you will learn about the origins of the project, the guiding principles used to cocreate and promote measurable, meaningful family engagement in systems-level change efforts, its goals and objectives, the impact of the coronavirus disease 2019 pandemic on the project and some high-level learnings from our experiences, which have implications for future care delivery improvements for this growing and vulnerable population of children.

What Families of Children With Medical Complexity Say They Need: Humanism in Care Delivery Change.

There is growing consensus that centering lived experience is needed to meaningfully transform the burdensome systems of care for children with medical complexity (CMC) and their families. The Collaborative Improvement and Innovation Network to Advance Care for Children with Medical Complexity quality improvement initiative, co-led with family colleagues, illuminates a critical real-world view of systems change to address unintended bias and demystify the medical model of care. We share candid themes in which families describe the need for systems to counteract widespread misconceptions and bias to achieve meaningful system change. We held family-designed, family-led focus groups (N = 127 across 27 groups) within 10 diverse state teams. Families were asked about CMC quality of life and family wellbeing. We transcribed and coded the responses to uncover salient themes. We uncovered 2 major themes from families with direct applicability to systems of care: "What's Missing - Human Dignity" and "What Families Really Need and Recommend in Care." Families shared that valuing each child and creating opportunities for the child and family to enjoy their lives were most important in addressing human dignity in systems of care. They recommended centering the whole child, building relationships of trust and communication, and valuing family-to-family supports to transform the system of care aligned to humanism in care. Families express an urgency for systems to uphold dignity, valuing their child as a whole human being whose quality of life holds meaning and joy, not just as a diagnosis. The highly untenable cost of navigating dehumanizing systems of care reduces quality of life and wellbeing and must be transformed.

Measuring What Matters to Children With Medical Complexity and Their Families.

The Collaborative Improvement and Innovation Network to Advance Care for Children with Medical Complexity (CMC CoIIN) was designed with a foundational commitment to partnership with family leaders to codesign and improve systems of care and supports for CMC and their families - this fundamental commitment was essential to the CMC CoIIN's measurement strategy. In this paper, we examine key learnings from partnering with family leaders from interdisciplinary state teams in the CMC CoIIN to identify and define quantitative quality improvement measures to improve care and support for CMC and their families, including quality of life, well-being, and flourishing; unmet health needs; and support systems such as medical home, patient and family engagement, and shared plans of care. Codesigning the CMC CoIIN measurement strategy with family leaders greatly enhanced our measurement approach and provided numerous unique learning opportunities for the CMC CoIIN's project team and state teams.

Defining and Promoting Pediatric Pulmonary Health: Equitable Family and Community Partnerships.

Optimizing pulmonary health across the lifespan begins from the earliest stages of childhood and requires a partnership between the family, pulmonologist, and pediatrician to achieve equitable outcomes. The Community Pediatrics session of the Defining and Promoting Pediatric Pulmonary Health workshop weaved together 4 community-based pillars with 4 research principles to set an agenda for future pediatric pulmonary research in optimizing lung and sleep health for children and adolescents. To address diversity, equity, and inclusion, both research proposals and workforce must purposefully include a diverse set of participants that reflects the community served, in addition to embracing nontraditional, community-based sites of care and social determinants of health. To foster inclusive, exploratory, and innovative research, studies must be centered on community priorities, with findings applied to all members of the community, particularly those in historically marginalized and minoritized groups. Research teams should also foster meaningful partnerships with community primary care and family members from study conceptualization. To achieve these goals, implementation and dissemination science should be expanded in pediatric pulmonary research, along with the development of rapid mechanisms to disseminate best practices to community-based clinicians. To build cross-disciplinary collaboration and training, community-academic partnerships, family research partnerships, and integrated research networks are necessary. With research supported by community pillars built on authentic partnerships and guided by inclusive principles, pediatric lung and sleep health can be optimized for all children and adolescents across the full lifespan in the community in which they live and thrive.

Outcomes of End-User Testing of a Care Coordination Mobile App With Families of Children With Special Health Care Needs: Simulation Study.

BACKGROUND: Care for children with special health care needs relies on a network of providers who work to address the medical, behavioral, developmental, educational, social, and economic needs of the child and their family. Family-directed, manually created visual depictions of care team composition (ie, care mapping) and detailed note-taking curated by caregivers (eg, care binders) have been shown to enhance care coordination for families of these children, but they are difficult to implement in clinical settings owing to a lack of integration with electronic health records and limited visibility of family-generated insights for care providers. Caremap is an electronic health record-integrated digital personal health record mobile app designed to integrate the benefits of care mapping and care binders. Currently, there is sparse literature describing end-user participation in the co-design of digital health tools. In this paper, we describe a project that evaluated the usability and proof of concept of the Caremap app through end-user simulation. OBJECTIVE: This study aimed to conduct proof-of-concept testing of the Caremap app to coordinate care for children with special health care needs and explore early end-user engagement in simulation testing. The specific aims included engaging end users in app co-design via app simulation, evaluating the usability of the app using validated measures, and exploring user perspectives on how to make further improvements to the app. METHODS: Caregivers of children with special health care needs were recruited to participate in a simulation exercise using Caremap to coordinate care for a simulated case of a child with complex medical and behavioral needs. Participants completed a postsimulation questionnaire adapted from 2 validated surveys: the Pediatric Integrated Care Survey (PICS) and the user version of the Mobile Application Rating Scale (uMARS). A key informant interview was also conducted with a liaison to Spanish-speaking families regarding app accessibility for non-English-speaking users. RESULTS: A Caremap simulation was successfully developed in partnership with families of children with special health care needs. Overall, 38 families recruited from 19 different US states participated in the simulation exercise and completed the survey. The average rating for the survey adapted from the PICS was 4.1 (SD 0.82) out of 5, and the average rating for the adapted uMARS survey was 4 (SD 0.83) out of 5. The highest-rated app feature was the ability to track progress toward short-term, patient- and family-defined care goals. CONCLUSIONS: Internet-based simulation successfully facilitated end-user engagement and feedback for a digital health care coordination app for families of children with special health care needs. The families who completed simulation with Caremap rated it highly across several domains related to care coordination. The simulation study results elucidated key areas for improvement that translated into actionable next steps in app development.

Advances in Hodgkin's lymphoma pharmacotherapy: a focus on histone deacetylase inhibitors.

INTRODUCTION: Classical Hodgkin lymphomas (cHL) usually have excellent cure rates. Yet, for patients with refractory or relapsed cHL, prognosis deteriorates as the disease becomes resistant to subsequent lines of therapies: autologous stem cell transplantation, brentuximab vedotin, and checkpoint inhibitors. Immune escape and drug resistance are hallmarks of Hodgkin Reed Sternberg cell survival, prompting the need for additional therapeutic strategies. Histone modification-based combination is an effective clinical strategy. AREAS COVERED: In this review, we discuss the different histone deacetylase (HDAC) inhibitor molecules that have been developed and studied in cancer therapy with a focus on cHL. We review their preclinical and clinical activities both as single agents and in combination studies. Literature search was conducted in PubMed, Google Scholar, and ClinicalTrials.gov databases, using search terms 'Hodgkin lymphoma,' 'histone deacetylase inhibitor', and variations on such (e.g. 'HDAC' and individual drug names) in combination using operators 'AND,' 'OR,' and 'NOT' according to Boolean logic. EXPERT OPINION: HDAC inhibitors alone will not be sufficient for the treatment of R/RcHL, but given their disease control capacity, synergistic interaction with currently approved drugs, and ability to overcome drug resistance, particularly PD-1 inhibitors, we believe that HDACinhibitors will eventually become incorporated into the treatment armamentarium of cHL.

Family-Educator Partnership in the Development of Entrustable Professional Activities in Complex Care.

PROBLEM: Incorporating patient and family voices in the development of entrustable professional activities (EPAs) is not standard practice. Care of children with medical complexity (CMC) is an area of pediatrics that relies on family partnership, and families of CMC are ideal partners in EPA development given their expertise in their child's care and experience interacting with the health care system. The authors describe their model for partnering with families to develop EPAs and reflect on the unique contributions of family leaders to the process. APPROACH: After recruitment of family leaders from a national organization of families and friends of children with special health care needs, the authors used a multistage process for EPA development from June 2019 to February 2021. Family leaders were integrated throughout the process, including creating EPA descriptions, revising content across all EPAs, appraising EPAs through virtual focus groups with other key stakeholders, and finalizing and publishing EPAs. The authors used content analysis to identify recommendations for patient- and family-integrated EPA development. OUTCOMES: Family leaders and educators partnered in every phase of developing EPAs for the care of CMC, including as content experts, editors, focus group facilitators, and coauthors. Family leaders recommended substantive changes to all EPAs, including revising language, augmenting content, and modifying scope of practice. In addition, content analysis of family leaders' revisions yielded 10 recommendations to ensure that written EPA descriptions are patient- and family-centered. NEXT STEPS: The described process of EPA development for the care of CMC models how families can be integrated into competency framework development and highlights their contributions. Family leader recommendations for embedding patient and family voices in EPA descriptions can serve as a guide for EPA development in other specialties.

Annual Days With a Health Care Encounter for Children and Youth Enrolled in Medicaid: A Multistate Analysis.

OBJECTIVE: To assess the number of days that children experienced a health care encounter and associations between chronic condition types and health care encounters. METHODS: Retrospective analysis of data from 5,082,231 children ages 0 to 18 years enrolled in Medicaid during 2017 in 12 US states contained in the IBM Watson Marketscan Medicaid Database. We counted and categorized enrollees' encounter days, defined as unique days a child had a health care visit, by type of health service. We used International Classification of Disease-10 diagnosis code categories from Agency for Healthcare Research and Quality's Chronic Condition Indicator System to identify chronic mental and physical health conditions. RESULTS: Median (interquartile range [IQR]) annual encounter days was 6 (2-13). Children in the 91st to 98th and ≥99th percentiles for encounter days experienced a median of 49 (IQR 38-70) and 229 (IQR 181, 309) days, respectively; these children accounted for 52.6% of days for the cohort. As encounter days increased from the 25th to >90th percentile, the percentage of children with co-existing mental and physical health conditions increased from <0.1% to 47.4% (P < .001). Outpatient visits accounted for a total of 68.3% and 62.2% of days for children the 91st to 98th and ≥99th percentiles. CONCLUSION: Ten percent of children enrolled in Medicaid averaged health care encounters at least 1 day per week; 1% experienced health care encounters on most weekdays. Further investigation is needed to understand how families perceive frequent health care encounters, including how to facilitate their children's care in the most feasible way.

Quality of Life and Well-Being for Children and Youth With Special Health Care Needs and their Families: A Vision for the Future.

OBJECTIVES: To fulfill the promise of a life of dignity, autonomy, and independence for children and youth with special health care needs (CYSHCN) and their families, greater value must be assigned to meaningful outcomes, such as quality of life and well-being. METHODS: Despite decades of research, programs, and measurements addressing quality of life and well-being for CYSHCN and their families, there still is no consensus on how to measure, implement, or achieve them. RESULTS: As the US health care system strives to reach the health care goals of safe, efficient, effective, equitable, timely, and patient-centered care, youth and families must be equal partners at all levels of the health care system-from clinical decision making to designing and implementing programs and policies. CONCLUSIONS: The health care system must systematically measure the priorities of CYSHCN and their families. It also must incorporate data on quality of life and well-being when developing services, supports, and systems that help CYSHCN and their families to flourish rather than hindering them.

PediHome: Development of a Family-Reported Measure of Pediatric Home Healthcare Quality.

OBJECTIVE: No validated tools exist to measure pediatric home healthcare quality. The objective of this work was to develop a family-reported survey (PediHome) to measure the quality of home healthcare for children with medical complexity (CMC). METHODS: A national multidisciplinary expert panel (N = 19) was convened to develop survey content domains. Panelist were joined by 3 additional experts to rank candidate survey items for importance and evaluate relevance and structure. Cognitive interviews were conducted with English-speaking (n = 12) and Spanish-speaking (n = 4) family caregivers of CMC to revise problematic items and clarify response options. A cross-sectional survey was then fielded (6/1/20-10/31/20) to parents whose children receive healthcare at 2 regional academic medical centers. RESULTS: The final measure included N = 28 total items with 4 items quantifying access, 1 evaluating overall quality rating, and 21 items assessing provider tasks (11 home nursing only, 2 certified nursing assistant/home health aide only, and 1 dual). Out of 312 caregivers of CMC, 142 (46%) responded and one-half (n = 68, 48%) reported a child receiving home nursing. They received a weekly median of 58.4% (IQR ±31.2%) of approved nursing hours with 55% reporting a missed nursing shift within the last month. Median overall quality was 75-9 (0-10 scale) and median scores on specific quality items ranged from 31-4 to 43-4 (0-4 scale). CONCLUSIONS: PediHome is a new content-valid family-reported measure of home healthcare quality for CMC that is useful for evaluating healthcare quality across several domains. Future work will involve assessing PediHome's construct and predictive validity.

A Multistakeholder Approach to the Development of Entrustable Professional Activities in Complex Care.

BACKGROUND: Care of children with medical complexity (CMC) involves unique knowledge, skills, and attitudes. Previous work has identified curricular priorities in complex care for pediatricians yet there are no established competency frameworks to guide curriculum development. We aimed to develop and appraise Entrustable Professional Activities (EPAs) for the care of CMC with multistakeholder involvement. METHODS: We recruited complex care practitioners to develop EPAs using a template for elaborating descriptive elements. A team of clinicians, educators, trainees, and family leaders refined EPAs and mapped content to the Accreditation Council for Graduate Medical Education Milestones. We conducted virtual focus groups to assess whether EPAs represented the essential skills of pediatricians caring for CMC. Focus group data were analyzed using content analysis. RESULTS: Content experts developed 11 EPAs for the care of CMC describing knowledge, skills, and attitudes required for attaining competency. EPAs were mapped to 21 of the 21 (100%) reporting pediatric milestones. Focus group participants endorsed and refined EPA content. Categories of feedback included clarifying medical knowledge, expanding on interpersonal communication skills, emphasizing systems-based practice, and affirming family partnership. CONCLUSIONS: A systematic approach to developing EPAs for the care of CMC provides a guide for curriculum development and assessment in complex care.

Moving From Spending to Investment: A Research Agenda for Improving Health Care Financing for Children and Youth With Special Health Care Needs.

Children and youth with special health care needs (CYSHCN) use disproportionately more health care resources than non-CYSHCN, and their unique needs merit additional consideration. Spending on health care in the United States is heavily concentrated on acute illnesses through fee-for-service (FFS). Payment reform frameworks have focused on shifting away from FFS, addressing health outcomes and the experience of care while lowering costs, particularly for high resource utilizers. The focus of payment reform efforts to date has been on adults with chronic illnesses, with less priority given to investment in children's health and life course. Spending for children's health is also considered an investment in their growth and development with long-term outcomes at stake, so research questions should focus on where and how such spending should be targeted. This paper discusses high-priority research topics in the area of health care financing for CYSHCN in the context of what is currently known and important knowledge gaps related to investment for CYSHCN. It proceeds to describe 3 potential research projects that can address these topics, following a framework informed by the priority questions identified in a previous multistakeholder research agenda development process. We focus on 3 areas: benefits, payment models, and quality measures. Specific aims and hypotheses are offered, as well as suggestions for approaches and thoughts on potential implications.

Local and distant tumor dormancy during early stage breast cancer are associated with the predominance of infiltrating T effector subsets.

BACKGROUND: Although breast cancer mortality is a result of distant recurrences associated with the establishment of tumor dormancy, current clinical practice guidelines recommend a wait and watch approach for tumor recurrences. This is because of our limited understanding of tumor dormancy and insufficient evidence in support of immunological control of tumor dormancy. METHODS: We used FVBN202 transgenic mice expressing rat neu oncogene in the mammary glands, and their parental FVB strain lacking neu expression. These models allowed the detection of tumor dormancy at distant sites using the rat neu protein as a tumor marker. We also used Ki67 for the detection of the indolent and quiescent types of tumor dormancy. Multicolor flow cytometry was used to detect dormant tumor cells and T cell subsets. Co-culture studies were performed to determine the role of T cells in preventing regrowth of dormant cells. RESULTS: We demonstrated that dormant tumor cells were present at the site of primary breast cancer and at distant sites in the lungs and in the liver very early in the course of early stage breast cancer when no distant metastasis was evident. Dormant tumor cells were characterized as neu expressing Ki67- and Ki67low fractions associated with the induction of local immune responses predominated by CD4+ and CD8+ T effector cell subsets. The presence of neu-autoreactive T cells from FVBN202 mice only prevented regrowth of dormant cells. On the other hand, presence of neu-alloreactive anti-tumor T cells in FVB mice prior to tumor challenge resulted in the protection of mice from the dissemination of dormant tumor cells to distant organs. CONCLUSION: Our results suggest that immunotherapeutic targeting of semi-allogeneic mutant neoantigens during tumor dormancy might prevent distant recurrence of the disease.

Pupillometry measures of autonomic nervous system regulation with advancing age in a healthy pediatric cohort.

PURPOSE: To determine if variables of the pupillary light response mature with age and sex in a healthy pediatric cohort and the utility of pupillometry in assessment among pediatric participants. METHODS: After 1 min in a dark room to establish baseline, pupillometry was performed on 323 healthy, pediatric participants (646 eyes; 2-21 years; 175 females). Variables included initial pupil diameter, pupil diameter after light stimulus, percent pupillary constriction, latency to onset of constriction, average constriction velocity, maximum constriction velocity, average dilation velocity, and time from light stimulus to 75% of the initial pupil diameter. Data analyses employed ANOVAs and non-linear regressions. RESULTS: Analyses of age group differences revealed that participants 12-21 years old had a larger initial pupil diameter and pupil diameter after light stimulus, with males aged 12-18 years demonstrating a larger pupil diameter than all younger participants (ps < 0.05). Participants 12-18 years old had a slower maximum constriction velocity than participants 6-11 years old, with no sex differences (ps < 0.05). Furthermore, males aged 12-18 years old had a smaller percent constriction than males 6-11 years old (ps < 0.05). Regressions revealed that percent constriction and dilation velocity seemed to mature linearly, initial pupil diameter and ending pupil diameter matured quadratically, and the constriction velocity terms matured cubically. CONCLUSIONS: Results revealed maturation of the pupillary light response by age and sex in healthy pediatric participants. Given the value of the pupillary light response as a biomarker, the results provide normative benchmarks for comparison in health and disease, including opiate-exposed and concussion patients.

Longitudinal studies can identify distinct inflammatory cytokines associated with the inhibition or progression of liver cancer.

BACKGROUND AND AIMS: Chronic diseases such as nonalcoholic fatty liver disease (NAFLD) and hepatocellular carcinoma (HCC) are associated with chronic inflammation. However, controversial reports as to the key cytokines involved in the process of chronic inflammation hinder development of targeted therapies for patients. This is because, chronic inflammatory process cannot be fully understood by studying the mechanisms of the disease in a short-term or isolated fashion. Understanding of the trend of inflammatory cytokines through longitudinal studies could provide a profound insight into the process of disease progression. METHODS: We performed a longitudinal analysis of inflammatory cytokines/chemokines and faecal microbiome dysbiosis associated with the diet-induced progression of NAFLD to HCC in diet-induced animal model of NAFLD comparing males and females, since males show a higher incidence of these diseases than females do. RESULTS: Longitudinal analyses revealed that a transient and timely increase in LIF and TMIP1 was associated with the inhibition of the progression of NAFLD to HCC in females. On the other hand, chronically increasing trends in CCL12, CCL17, CXCL9 and LIX/CXCL5 were associated with the promotion of the progression of NAFLD to HCC in males. CONCLUSIONS: We provided empirical evidence that a methodological shift from snapshot observations to longitudinal data collection and analysis can provide a better understanding of chronic liver diseases.

Empowered by Nurses.

Before Justice Hope Coleman was born, nurses in an antenatal testing center provided her parents with support and encouragement, the first steps in empowering them in their roles as parents of a child with multiple disabilities and complex medical needs. Over time, other nurses supported Justice's parents as they learned to communicate clearly and collaboratively with professionals; to provide a high level of care for their daughter in a loving, family-oriented way; and to advocate not only for Justice and their family as a whole, but also for other children and families in the healthcare system. This article describes the important role nurses played in empowering Justice's parents.